A Safety, Tolerability, and Efficacy Study of VX-264 in Subjects With Type 1 Diabetes

Phase 1

Conditions: Type 1 Diabetes Mellitus
MedDRA version: 20.0Level: PTClassification code 10012601Term: Diabetes mellitusSystem Organ Class: 10027433 - Metabolism and nutrition disorders
Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Registration Number: EUCTR2022-003318-35-DE

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 23

Inclusion Criteria

1. Subjects (male and female) between the ages of 18 and 65 years (inclusive) on the date of first informed consent.
2. HbA1c =6.0% and =9.5%.
3. Clinical history and laboratory evidence of T1D based on the American Diabetes Association/European Association for the Study of Diabetes algorithm 13 for investigation of suspected T1D including:
o insulin dependence for =5 years at time of Screening, and
o peak stimulated C-peptide level during MMTT <50 pmol/L (<0.15 ng/mL)
4. Consistent use of CGM for at least 4 weeks before Screening and willingness to use only the study-provided CGM for the duration of the study.
5. Body habitus supportive of implantation of the planned number of VX-264 units per study part and/or cohort.
Please refer to Section 8.1 of the Protocol for additional inclusion criteria.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 21
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

1. Prior islet cell transplant, organ transplant, or cell therapy.
2. Advanced complications associated with diabetes including untreated proliferative retinopathy, diabetic nephropathy, skin ulcers, or amputations attributable to diabetes.
3. Insulin requirement:
a) Parts A and B >40 U/day, or <10 U/day
b) Part C: >55 U/day, or <10 U/day
c) >0.8 U/kg/day.
4. Subjects with =2 or more episodes of severe hypoglycemia in the 12 months prior to signing of informed consent at Screening.
5. Previous abdominal or abdominal wall surgery, or history of peritonitis, that can impact VX-264 placement or put the patient at higher risk of surgical complications
Please refer to Section 8.2 of the Protocol for additional exclusion criteria.